Reduced hemolysis, lowered thrombosis rate and improved quality of life in patients with PNH who had previously never been transfused
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Alexion has presented new data, which demonstrates the severe clinical burden of paroxysmal nocturnal hemoglobinuria (PNH) and the clinical benefits of Soliris (eculizumab) in 44 patients with no history of prior blood transfusions. Prior to Soliris treatment, these never-transfused patients with PNH demonstrated increased intravascular hemolysis, 87% reported impaired quality of life, and 28% had a history of clinically evident thrombosis.
Soliris, a first-in-class terminal complement inhibitor, has shown to reduced hemolysis in all patients assessed in the retrospective study. 91% of patients with quality of life assessments both before and during Soliris treatment showed improvement in their quality of life during Soliris treatment. Researchers also observed a significant reduction in clinically evident thrombosis, from 7.85 events per 100 patient years to zero events (p<0.001) following Soliris treatment.
The data were presented at the 51st Annual Meeting of the American Society of Hematology (ASH) in a poster titled, ‘Clinical Impact of Unregulated Terminal Complement Activity in Never-Transfused Patients with Paroxysmal Nocturnal Hemoglobinuria.’
Leonard Bell, chief executive officer of Alexion, said: “This study shows both the central role of unregulated complement activity and chronic hemolysis in PNH, and the significant clinical benefits of Soliris in all patients with PNH, regardless of pre-treatment transfusion status or hemoglobin levels.”
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